Premarketing and Clinical Development Flashcards

Which IND type allows a sponsor to ship an investigational drug to clinical investigators without a 30-day FDA review period?

Answer: Emergency IND

An Emergency IND allows FDA to authorize use over the phone before submission, bypassing the standard 30-day waiting period.

In a Phase I dose-escalation oncology trial, what is the primary endpoint typically used to determine the Maximum Tolerated Dose (MTD)?

Answer: Dose-Limiting Toxicity (DLT)

DLTs observed during the first treatment cycle define the MTD boundary in classic 3+3 and modified dose-escalation designs.

Under 21 CFR Part 312, which section requires sponsors to report unexpected serious adverse events from foreign clinical studies to the IND within 15 calendar days?

Answer: 312.32

21 CFR 312.32 governs IND safety reporting, requiring 15-day expedited reports for unexpected serious adverse drug reactions from any source.

A sponsor wants to conduct a single-dose PK study in six healthy volunteers using a microdose (≤100 mcg) to characterize absorption without triggering full IND requirements. Which IND category applies?

Answer: Exploratory IND

Exploratory INDs (Phase 0) permit very limited human exposure with microdoses primarily to obtain early pharmacokinetic or pharmacodynamic data.

What is the purpose of a Special Protocol Assessment (SPA) agreement between a sponsor and FDA for a Phase III trial?

Answer: To secure FDA agreement that the protocol design is acceptable to support a future NDA/BLA

An SPA provides sponsors with FDA's binding agreement that the protocol design, endpoints, and statistical analysis plan are acceptable for regulatory approval.

Which ICH guideline addresses the timing and extent of carcinogenicity studies needed to support clinical development of pharmaceuticals?

Answer: ICH S1A

ICH S1A provides recommendations on the need for carcinogenicity studies, specifying criteria based on treatment duration and patient population.

During clinical development, a sponsor discovers that an investigational product lot used in an ongoing trial fails a stability specification. What is the immediate regulatory obligation?

Answer: Report as an IND safety report if the failure poses a risk to subjects

If a stability failure creates a reasonable possibility of risk to subjects, it must be reported as an IND safety report under 21 CFR 312.32.